- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
- Industry
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 73–96
of 140
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
- Interventions
- Abatacept
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
- Interventions
- Stem Cell Transplant, Myeloablative conditioning regimen
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2015
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Interventions
- umbilical cord blood transplantation
- Procedure
- Lead sponsor
- Herbert Irving Comprehensive Cancer Center
- Other
- Eligibility
- Up to 60 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1997
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- placebo (saline), Olipudase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2023
- U.S. locations
- 3
- States / cities
- San Francisco, California • Decatur, Georgia • The Bronx, New York
- Conditions
- Hematopoietic and Lymphatic System Neoplasm, Histiocytic Sarcoma, Malignant Histiocytosis, Peripheral T-Cell Lymphoma, Not Otherwise Specified, Primary Cutaneous T-Cell Non-Hodgkin Lymphoma, Recurrent Chronic Lymphocytic Leukemia, Recurrent Chronic Myelomonocytic Leukemia, Recurrent Follicular Lymphoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoplasmacytic Lymphoma, Recurrent Myelodysplastic Syndrome, Recurrent Myelofibrosis, Recurrent Small Lymphocytic Lymphoma, Recurrent Waldenstrom Macroglobulinemia, Refractory Chronic Lymphocytic Leukemia, Refractory Chronic Myelomonocytic Leukemia, Refractory Erdheim-Chester Disease, Refractory Follicular Lymphoma, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoplasmacytic Lymphoma, Refractory Myelodysplastic Syndrome, Refractory Myelofibrosis, Refractory Small Lymphocytic Lymphoma, Refractory Waldenstrom Macroglobulinemia, Rosai-Dorfman-Destombes Disease, Primary Central Nervous System Lymphoma, Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm, Recurrent Erdheim-Chester Disease, Recurrent Fibroblastic Reticular Cell Sarcoma, Recurrent Histiocytic Sarcoma, Recurrent Interdigitating Dendritic Cell Sarcoma, Recurrent Rosai-Dorfman-Destombes Disease, Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm, Refractory Fibroblastic Reticular Cell Sarcoma, Refractory Histiocytic Sarcoma, Refractory Interdigitating Dendritic Cell Sarcoma, Refractory Rosai-Dorfman-Destombes Disease
- Interventions
- Axl/Mer/CSF1R Inhibitor Q702, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography, Survey Administration
- Drug · Procedure · Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 2
- States / cities
- Scottsdale, Arizona • Rochester, Minnesota
- Conditions
- Langerhans Cell Histiocytosis
- Interventions
- Clofarabine
- Drug
- Lead sponsor
- Dana-Farber Cancer Institute
- Other
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2026
- U.S. locations
- 14
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 8 more
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Erdheim-Chester Disease
- Interventions
- Not listed
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin
- Drug
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • Morristown, New Jersey
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
- Interventions
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2026
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Castleman's Disease (CD), Langerhans Cell Histiocytosis (LCH), Non-Langerhans-Cell Histiocytosis
- Interventions
- Genetic testing
- Diagnostic Test
- Lead sponsor
- Case Comprehensive Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Supportive Care Needs of Caregivers
- Interventions
- online surveys
- Behavioral
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 250 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Niemann-Pick Diseases
- Interventions
- Not listed
- Lead sponsor
- National Niemann-Pick Disease Foundation
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Neimann-Pick Disease
- Interventions
- Vorinostat
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Langerhans Cell Histiocytosis
- Interventions
- GSK2110183
- Drug
- Lead sponsor
- GlaxoSmithKline
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Baltimore, Maryland • Boston, Massachusetts • New York, New York + 2 more
- Conditions
- Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Acute Myeloid Leukemia, Chronic Myelogenous Leukemia, Non Hodgkin Lymphoma, Hemophagocytic Lymphohistiocytosis, Familial Hemophagocytic Lymphohistiocytosis, Hemophagocytic Syndrome, Epstein Barr Virus Infection, X-linked Lymphoproliferative Disease
- Interventions
- iCaspase9-transduced T cells, AP1903
- Biological · Drug
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2029
- U.S. locations
- 2
- States / cities
- Houston, Texas
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- Olipudase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Primary Haemophagocytic Lymphohistiocytosis
- Interventions
- NI-0501
- Biological
- Lead sponsor
- Swedish Orphan Biovitrum
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 8
- States / cities
- Aurora, Colorado • Wilmington, Delaware • Atlanta, Georgia + 5 more
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneuronal Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
- Interventions
- dabrafenib, trametinib
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 1 Year to 99 Years
- Enrollment
- 163 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 11
- States / cities
- Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl Cysteine
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 12 Months and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
- Interventions
- Hematopoietic Stem Cell Transplant
- Biological
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 4 Months to 45 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
- Conditions
- Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease, Other RAS/RAF/MEK/ERK Mutated Tumors
- Interventions
- HH2710
- Drug
- Lead sponsor
- Haihe Biopharma Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 2
- States / cities
- Lafayette, Indiana • Detroit, Michigan
- Conditions
- Cancer
- Interventions
- Trametinib, Dabrafenib
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 1 Month to 17 Years
- Enrollment
- 139 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • San Francisco, California • Washington D.C., District of Columbia + 7 more